After reviewing the safety data from two cohorts in a clinical trial testing the safety and effectiveness of escalating doses of AST-OPC1 stem cells in patients with spinal cord injury (SCI), the Data Monitoring Committee (DMC) from Asterias Biotherapeutics Inc has given its OK for the study to continue in a third cohort.
Dubbed the SCiStar study, the Phase 1/2a clinical trial, which is funded in part by a $14.3 million grant from the California Institute for Regenerative Medicine, is designed to evaluate the safety and effectiveness of Asterias’ AST-OPC1 cells among newly injured patients with sensory and motor complete cervical SCI (AIS-A patients) as well as newly injured patients with sensory incomplete SCI (AIS-B patients).
The initial cohort of three patients was dosed with 2 million cells, and the second cohort of five patients was dosed with 10 million cells. The recent approval from the DMC clears Asterias to now begin dosing a third cohort of five to eight AIS-A patients with the highest dose of 20 million cells, according to a media release from Fremont, Calif-based Asterias Biotherapeutics.
“The positive safety data in the previous phase 1 study and in the ongoing Phase 1/2a study gives us the confidence to now proceed to administration of 20 million cells, which based on our significant pre-clinical research is likely well within the dosing range where we would expect to see clinically meaningful improvement in these patients,” says Dr Edward Wirth, chief medical officer of Asterias Biotherapeutics, in the release.
A media release from CIRM explains that the AST-OPC1 cells are a form of cells called oligodendrocyte progenitors, which are capable of becoming several different kinds of cells. Some of which play a supporting role and help protect nerve cells in the central nervous system, including areas of the spinal cord that are damaged in spinal cord injury.
The patients enrolled in the trial have experienced injuries in the C5-C7 vertebrae and have essentially lost all feeling and movement below the injury site, with severe paralysis of the upper and lower limbs. They are treated with the cells 14 to 30 days after the injury was sustained, per the release.
“Our focus is always on the patient, so making sure a potential therapy is safe is an important first step,” states C. Randal Mills, PhD, the president and CEO of CIRM, in the release.
“I recently met with Jake Javier, a young man who was treated in this trial, and heard first-hand what he and his family are going through in the aftermath of his injury. But I also saw a young man with remarkable courage and determination. It is because of Jake, and the others who volunteer to take part in clinical trials, that progress is possible. They are true heroes,” he adds.
[Source(s): Asterias Biotherapeutics Inc, California Institute for Regenerative Medicine, Science Daily]